Abeona Therapeutics announced the appointment of Joao Siffert, M.D. as CEO, effective immediately. Siffert joined Abeona as Head of Research & Development and Chief Medical Officer in 2018, and has served as interim CEO since November of last year. Siffert will retain his responsibilities as Head of R&D and CMO until a clinical development lead is identified.

Abeona Therapeutics announced that it has appointed Christine Silverstein to the position of CFO and named Edward Carr as Chief Accounting Officer. Silverstein was previously Senior Vice President, Finance and Investor Relations for Abeona. Carr most recently served as Vice President and Assistant Controller at Coty (COTY).

Abeona Therapeutics announced key pipeline updates during the company's 2018 R&D Day. The company expects to initiate a pivotal clinical trial evaluating the potential of EB-101 for the treatment of RDEB in the middle of 2019. The VITAL Study will be a multicenter, randomized, Phase III clinical trial assessing 10-15 patients treated with EB-101 compared to intra-patient untreated wounds. The primary outcome measure of the study will be the proportion of treated wounds with greater than50% healing at three months, with secondary endpoints of investigator global assessment of wounds and changes in pain and itch from baseline. The company also reported that it has established GMP manufacturing capability for EB-101 at its gene therapy manufacturing facility in Cleveland. The facility, known as the Elisa Linton Center for Rare Disease Therapies, can produce clinical product, and has scalable capacity to support the potential commercial launch of EB-101. The company plans to amend its ongoing Phase I/II trial evaluating ABO-102 for MPS IIIA to enroll patients at earlier stages of disease. ABO-102 has been well tolerated to date with no serious drug-related adverse events. The study has also demonstrated a substantial, dose-related improvement in biomarkers, including reductions in cerebrospinal fluid heparan sulfate levels and liver volume in patients treated with ABO-102. Investigators also observed encouraging neurocognitive signals in younger, higher functioning patients enrolled in the higher dose of Cohort 3. Patients unable to participate in the modified Phase I/II study may be eligible to enroll in other studies within our MPS IIIA program. The company presented new preclinical data that will inform the submission of an investigational new drug application for ABO-202 in the first quarter of 2019. Findings from a combination pre-clinical, dose-escalation study suggest that ABO-202 may have a favorable safety profile, with no significant toxicology findings. Other IND-enabling studies also demonstrated normalized survival, improvement of motor function and cognition in affected mice treated with ABO-202, and that combination dosing of intravenous and intrathecal administrations may enhance the therapeutic potential of ABO-202.